Cogent Biosciences, Inc. (COGT) is a biotechnology company focused on developing precision therapies for genetically defined diseases. The company's approach is to design rational precision therapies that treat the underlying cause of disease and improve the lives of patients. Cogent's most advanced program is bezuclastinib, also known as CGT9486, a highly selective tyrosine kinase inhibitor designed to potently inhibit the KIT D816V mutation as well as other mutations in KIT exon 17.
Business Overview
Cogent's pipeline consists of bezuclastinib, which is being developed for the treatment of Systemic Mastocytosis (SM) and Gastrointestinal Stromal Tumors (GIST), as well as a portfolio of novel targeted therapies targeting mutations in FGFR2, ErbB2, and PI3Kα. The company's research team in Boulder, Colorado is focused on pioneering best-in-class, small molecule therapeutics to expand its pipeline and deliver novel precision therapies for patients living with unmet medical needs.Bezuclastinib for Systemic Mastocytosis (SM)
The vast majority of Advanced Systemic Mastocytosis (AdvSM) and Non-Advanced Systemic Mastocytosis (Non-AdvSM) patients have a KIT D816V mutation. Patients with AdvSM have a significantly diminished lifespan with a median survival of less than 3.5 years, while patients with Non-AdvSM suffer from a poor quality of life. The FDA has granted orphan drug designation to bezuclastinib for the treatment of Mastocytosis.Cogent is developing bezuclastinib in two key clinical trials: 1. SUMMIT (Non-AdvSM): A randomized, global, multicenter, double-blind, placebo-controlled, multi-part Phase 2 clinical trial for patients with Non-AdvSM. SUMMIT Part 1 completed enrollment in Q3 2023, including over-enrollment at 54 patients across Part 1a and Part 1b. SUMMIT Part 2 is a registration-directed study that is currently enrolling and expected to include 159 patients. Cogent is on-track to complete enrollment in Q2 2025, with top-line results expected by the end of 2025.
2. APEX (AdvSM): A global, open-label, multi-center, Phase 2 clinical trial in patients with AdvSM. Cogent initiated the registration-directed Part 2 of the APEX trial at a 150 mg daily dose of bezuclastinib in April 2023 and is on-track to complete enrollment by the end of 2024, with top-line results expected by mid-2025.
Bezuclastinib for Gastrointestinal Stromal Tumors (GIST)
Cogent is also pursuing the development of bezuclastinib in combination with sunitinib as a potential second-line treatment for patients living with GIST. GIST is a cancer frequently driven by KIT mutations, and resistance to currently available therapeutics is frequently associated with the emergence of other KIT mutations.PEAK is Cogent's randomized open-label, global Phase 3 clinical trial designed to evaluate the safety, tolerability, and efficacy of bezuclastinib in combination with sunitinib compared to sunitinib alone in patients with locally advanced, unresectable or metastatic GIST who have received prior treatment with imatinib. The FDA and EMA have granted orphan drug designation to bezuclastinib for the treatment of GIST. Cogent is on-track to complete enrollment in PEAK by the end of 2024, with top-line results expected by the end of 2025.
Research Programs
In addition to bezuclastinib, Cogent's research team is developing a portfolio of novel targeted therapies to help patients fighting serious, genetically driven diseases. The team's initial efforts are aimed at targeting currently undrugged mutations in fibroblast growth factor receptor (FGFR), ErbB2, and PI3Kα.Financials
For the full year 2023, Cogent reported an annual net loss of $192.4 million, with no revenue generated. The company's annual operating cash flow was -$153.6 million, and its annual free cash flow was -$156.4 million.In the first quarter of 2024, Cogent reported a net loss of $58.3 million. Research and development expenses were $52.7 million, up from $36.0 million in the prior year period, driven by increased costs associated with the advancement of the company's clinical programs. General and administrative expenses were $9.7 million, up from $7.2 million in the prior year period, primarily due to higher personnel costs.
As of March 31, 2024, Cogent had cash, cash equivalents, and marketable securities of $435.7 million, which the company believes will be sufficient to fund its operating expenses and capital expenditure requirements into 2027 and through clinical readouts from the ongoing SUMMIT, PEAK, and APEX registration-directed trials.